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Current Medicinal Chemistry

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ISSN (Print): 0929-8673
ISSN (Online): 1875-533X

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Perspective

Mitapivat: A Novel Treatment of Hemolytic Anemia in Adults with Pyruvate Kinase Deficiency

Author(s): Surya K. De*

Volume 31, Issue 6, 2024

Published on: 26 May, 2023

Page: [683 - 687] Pages: 5

DOI: 10.2174/0929867330666230430002709

Abstract

Mitapivat is an orally bioavailable small molecule allosteric activator of pyruvate kinase. It was approved by the US FDA on February 17, 2022, and the European Union in November 2022 for the treatment of hemolytic anemia in adult patients with pyruvate kinase deficiency. In this short perspective, physicochemical properties, synthesis, dosage and administration, mechanism of action, pharmacodynamics, pharmacokinetics, drug interactions, and adverse reactions of mitapivat are described.

Keywords: Hemolytic anemia, pyruvate kinase deficiency, mitapivat, sickle cell disease, thalassemia, red blood cells, hemoglobin.

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